Treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) With 3, 4 DAP

Treatment of Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenic Syndromes With 3, 4-Diaminopyridine

Lambert Eaton Myasthenic Syndrome (LEMS) is rare neurological disorder that results in muscle weakness and limited reflex activity. More than half of LEMS cases are associated with a malignancy, usually small cell lung cancer, and tend to progress more quickly than cases not coupled with malignant cells.

3,4diaminopyridine (3,4DAP)is a drug that has been demonstrated to be effective in treating the weakness associated with LEMS as it increases strength and improves autonomic symptoms in LEMS patients. It is not currently approved by the FDA for use in the United States. The investigators plan to use 3,4DAP to treat patients with LEMS here at the Columbia University MDA/ALS Research Center.

Study Overview

• Status: No longer available
• Conditions: Lambert Eaton Myasthenic Syndrome (LEMS)
• Intervention / Treatment: Drug: 3,4-diaminopyridine

• Study Type: Expanded Access

Contacts and Locations

Study Locations

• United States
  • New York
    • New York, New York, United States, 10032
      • Columbia University Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Be diagnosed with LEMS or a type of CMS likely to respond to 3, 4-DAP.
• If female of childbearing age, have negative pregnancy test, and be willing to practice and effective form of birth control during the study.
• Tested and found by ECG not to have a prolonged QTc syndrome.
• Agrees to have a second ECG at the time of peak drug effect. Has understood and signed the Informed Consent.

Exclusion Criteria:

• Is known to have a sensitivity to 3, 4-DAP.
• Has a history of past or current seizures or of severe asthma, or has an epileptiform EEG.
• Is believed by the investigator to be unable to comply with the protocol.
• Is unable to give informed consent.
• No patient will be excluded based on race, ethnicity, gender, or HIV status

Study Plan

How is the study designed?

Collaborators and Investigators

• Sponsor: Louis H. Weimer, MD
• Investigators: Principal Investigator: Louis H Weimer, MD, Columbia University

Study record dates

Study Major Dates

• Study Start: May 1, 2005
• Primary Completion (Anticipated): May 1, 2015

Study Registration Dates

• First Submitted: June 20, 2011
• First Submitted That Met QC Criteria: June 21, 2011
• First Posted (Estimate): June 22, 2011

Study Record Updates

• Last Update Posted (Estimate): July 18, 2013
• Last Update Submitted That Met QC Criteria: July 17, 2013
• Last Verified: July 1, 2013

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Neoplasms; Autoimmune Diseases of the Nervous System; Autoimmune Diseases; Neoplasms by Site; Disease; Neuromuscular Diseases; Neurodegenerative Diseases; Nervous System Neoplasms; Paraneoplastic Syndromes, Nervous System; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Myasthenia Gravis; Syndrome; Lambert-Eaton Myasthenic Syndrome; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Peripheral Nervous System Agents; Membrane Transport Modulators; Neuromuscular Agents; Potassium Channel Blockers; Amifampridine
• Other Study ID Numbers: AAAB2528